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Efficacy and Safety of Subcutaneous Dupilumab in Participants With Asthma/Asthmatic Wheeze Aged 2 to <6 Years (LIBERTY ASTHMA TREKIDS)

Phase 3
Recruiting
Conditions
Wheezing
Asthma
Interventions
Drug: Placebo
Drug: Dupilumab
Registration Number
NCT06191315
Lead Sponsor
Sanofi
Brief Summary

This is a parallel, Phase 3, 2-arm study to evaluate the efficacy and long-term safety of dupilumab treatment in children 2 to \<6 years of age with uncontrolled asthma and/or recurrent severe asthmatic wheeze. The study will be conducted in 2 parts. Part A will be a 52-week, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of dupilumab in children aged 2 to \<6 years old with uncontrolled asthma and/or recurrent severe asthmatic wheeze. At the end of Part A, all eligible participants will be offered participation in Part B, an optional open-label extension phase.

Study details include:

Part A:

The study duration of part A will be up to 68 weeks consisting of a 4-week Screening, a 52week treatment period, and a 12-week post-treatment follow-up period. For participants who will chose to participate in Part B, the study duration will be up to 120 weeks (additional 52-week treatment period).

Part B:

For participants who will choose to participate in Part B, the study duration will be up to 120 weeks (Part A \[4-week Screening and a 52-week treatment period\] plus additional 52-week treatment period and a 12-week post-treatment follow-up period).

Detailed Description

The duration per participant is up to 120 Weeks.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
90
Inclusion Criteria
  • Participant must be 2 to <6 years of age

  • Diagnosis of asthma or recurrent severe asthmatic wheeze that is not controlled with chronic ICS for at least 3 months with stable use of at least low dose ICS for ≥1 month prior to Screening Visit 1 with evidence of uncontrolled asthma and/or recurrent severe asthmatic wheeze*.

  • At least one additional major criterion from the modified asthma predictive index:

    1. Physician diagnosed Atopic Dermatitis,

    2. Allergic sensitization to at least 1 aeroallergen (with a positive serum IgE defined as a value ≥0.35 kU/L).

      OR 2 minor criteria:

    3. Wheezing unrelated to colds,

    4. Peripheral blood eosinophilia ≥4%,

    5. Allergic sensitization to milk, eggs, or peanuts (defined by serum specific IgE >0.35 kU/L.

  • Parent(s)/caregiver(s)/legal guardian(s) willing and able to comply with clinic visits and study-related procedures.

  • Parent(s)/caregiver(s)/legal guardian(s) able to understand the study requirements.

  • Participants/parent(s)/caregiver(s)/legal guardian(s), as appropriate, must be able to understand and complete study-related questionnaires

  • Body weight at screening and randomization >5 kg and <30 kg.

  • Parents or caregivers or legal guardian capable of giving signed informed consent.

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Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply:

  • Severe asthma with the need for chronic oral/systemic corticosteroid use (>1 month continuous) at the time of screening enrollment.
  • History of a systemic hypersensitivity reaction or anaphylaxis to dupilumab or any other biologic therapy, including any excipient.
  • History of prematurity (<34 weeks gestation).
  • Any other chronic lung disease that would impair lung function (eg, cystic fibrosis, bronchopulmonary dysplasia) or chronic lung disease of prematurity or need for oxygen for more than 5 days in the neonatal period.
  • History of life-threatening asthma (eg, requiring intubation).

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboPlacebo matching dupilumab subcutaneous injection as per protocol
Dupilumab (double-blind period)DupilumabDupilumab subcutaneous injection as per protocol
Primary Outcome Measures
NameTimeMethod
Part A: Annualized rate of severe asthma exacerbations during the 52-week treatment periodBaseline through Week 52

Annualized rate of severe asthma exacerbations during the 52-week treatment period.

Part B: Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), and AEs leading to permanent treatment discontinuationWeek 52 through Week 116

Incidence of TEAEs, SAEs, AESIs, and AEs leading to permanent treatment discontinuation.

Secondary Outcome Measures
NameTimeMethod
Part A: IgG response to nonlive vaccinationBaseline through Week 52
Part A: Annualized rate of hospitalization, ER or urgent care visit for asthma exacerbation during the 52week treatment period.Baseline through Week 52
Part A: Physician Global Assessment of Change of the child's asthma control (PGA-change in asthma control) at Week 52Week 52

The Physician Global Assessment of Change of Asthma (PGA-change in asthma control) is a 1 item questionnaire that asks the physician to provide the overall assessment of change in the child's asthma control on a 7-point scale, compared to just before he/she started taking the study treatment. Response choices are: 0 = "Very much better", 1 = "Moderately better", 2 ="A little better", 3 = "No change", 4 = "A little worse", 5 = "Moderately worse", 6 = "Very much worse".

Part A: Mean number of days without asthma symptoms (DWAS) using the Pediatric Asthma Caregiver Diary (PACD) during the 52-week treatment periodBaseline through Week 52

A day without asthma symptoms (DWAS) is defined as a day without any asthma symptoms, β-agonist use, oral steroid use, or need for urgent asthma care. The PACD consists of a daytime and overnight record. The daytime asthma symptom questions are scored on a 6-point scale from 0 (no symptoms/interference) to 5 (very severe symptoms/interference). The overnight questions are scored on a 5-point scale from 0 (no overnight cough and caregiver not disturbed at all) to 4 (coughed all night and disturbed all night).

Part A: Change from baseline to Week 52 in Test for Respiratory and Asthma Control in Kids (TRACK)Baseline to Week 52

TRACK is a validated questionnaire for caregiver completion for preschool aged children with symptoms consistent with asthma. The total score range of 0 to 100. Higher scores indicated better asthma control.

Part A: Change from baseline to Week 52 in Caregiver Global Impression of their child's asthma control (CGI-asthma control)Baseline to Week 52

The Caregiver Global Impression of asthma control (CGI-Asthma Control) is a 1-item questionnaire that asks caregiver to provide the assessment of their child's asthma control over the past 7 days, on a 4-point scale for the past week. Response choices are: 1 = "Not controlled", 2 = "Somewhat controlled", 3 = "Moderately controlled", 4 = "Well controlled".

Part A: Change from baseline to Week 52 in Caregiver Global Impression of their child's asthma severity (CGI-asthma severity)Baseline to Week 52

The Caregiver Global Impression of asthma severity (CGI-Asthma Severity) is a 1-item questionnaire that asks caregivers to provide the assessment of their child's asthma control over the past 7 days, on a 4-point scale for the past week. Response choices are: 1 = "None", 2 = "Mild", 3 = "Moderate", 4 = "Severe".

Part A: Change from baseline to Week 52 in Physician Global Assessment of the child's asthma control (PGA-asthma control).Baseline to Week 52

The Physician Global Assessment of asthma control is a 1-item questionnaire that asks physicians to assess the control of the child's asthma in the past 7 days on a 4 point scale for the past week. Response choices are: 1 = "Not controlled", 2 = "Somewhat controlled", 3 = "Moderately controlled", 4 = "Well controlled".

Part A: Change from baseline in blood eosinophil level at Weeks 24 and 52Baseline to Week 24 and 52
Part A: Annualized rate of moderate asthma exacerbations during the 52-week treatment periodBaseline through Week 52

Annualized rate of moderate asthma exacerbations during the 52-week treatment period.

Part A: Cumulative ICS dose during the 52-week treatment periodBaseline through Week 52
Part A: Change from baseline to Week 52 in daytime symptom score using the daytime record of PACDBaseline to Week 52

The PACD consists of a daytime and overnight record. The daytime asthma symptom questions are scored on a 6-point scale from 0 (no symptoms/interference) to 5 (very severe symptoms/interference).

Part A: Incidence of TEAEs, SAEs, AESIs, and AEs leading to permanent treatment discontinuationBaseline through Week 52
Part A: Change from baseline to Week 52 in Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic ScaleBaseline to Week 52

PedsQL measures health-related quality of life (HRQOL) in healthy children and adolescents and those with acute and chronic health conditions. Scores range 0-100 with higher scores indicate better HRQOL.

Part A: Caregiver Global Impression of Change in their child's asthma control (CGI-change in asthma control) at Week 52Week 52

CGI-change in asthma control is a 1-item questionnaire that asks the caregiver to provide the overall assessment of change of their child's asthma control on a 7-point scale, compared to just before he/she started taking the study treatment. Response choices are: 0 = "Very much better", 1 ="Moderately better", 2 = "A little better", 3 = "No change", 4 = "A little worse", 5 = "Moderately worse", 6 = "Very much worse".

Part A: Change from baseline in weekly average use of reliever medication during the 52-week treatment periodBaseline to Week 52
Part A: Concentration of dupilumab in serum over time during the 52-week treatment periodBaseline through Week 52
Part A: Incidence of treatment-emergent anti-drug antibody (ADA) against dupilumab over timeBaseline through Week 52
Part B: Annualized rate of severe asthma exacerbations events during the 52-week Part B treatment periodWeek 52 through Week 104

Annualized rate of severe asthma exacerbations events during the 52-week Part B treatment period.

Part B Concentration of dupilumab in serum over time during the 52-week Part B treatment periodWeek 52 through Week 104
Part B: Incidence of treatment-emergent anti-drug antibodies (ADAs) against dupilumab over timeWeek 52 through Week 104

Trial Locations

Locations (58)

Investigational Site Number : 3480005

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Budapest, Hungary

Investigational Site Number : 3480004

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Debrecen, Hungary

Investigational Site Number : 3480001

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Szigetvár, Hungary

Investigational Site Number : 3480002

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Székesfehérvár, Hungary

Buzzi Children's Hospital - Investigational Site Number : 3800003

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Milan, Lombardia, Italy

Investigational Site Number : 2500003

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Paris, France

Investigational Site Number : 2760002

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Düsseldorf, Germany

Investigational Site Number : 2760001

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Leipzig, Germany

Investigational Site Number : 3000001

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Athens, Greece

Phoenix Children's Hospital- Site Number : 8400001

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Phoenix, Arizona, United States

Newport Children's Medical Group - Newport Beach- Site Number : 8400023

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Newport Beach, California, United States

Allervie Health - Destin (AllerVie Clinical Research)- Site Number : 8400016

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Destin, Florida, United States

Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400011

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Chicago, Illinois, United States

Allergy and Asthma Specialist- Site Number : 8400002

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Owensboro, Kentucky, United States

Mayo Clinic Hospital Rochester- Site Number : 8400008

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Rochester, Minnesota, United States

UBMD Pediatrics- Site Number : 8400013

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Buffalo, New York, United States

Boston Children's Health Physicians - Hawthorne- Site Number : 8400010

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Hawthorne, New York, United States

UNC Children's Hospital- Site Number : 8400005

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Chapel Hill, North Carolina, United States

Cincinnati Children's Hospital Medical Center- Site Number : 8400004

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Cincinnati, Ohio, United States

University Hospitals Cleveland Medical Center- Site Number : 8400015

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Cleveland, Ohio, United States

Vanderbilt University Medical Center- Site Number : 8400024

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Nashville, Tennessee, United States

South Texas Medical Research Institute - TTS Research- Site Number : 8400022

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Boerne, Texas, United States

Investigational Site Number : 0320008

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Buenos Aires, Ciudad De Buenos Aires, Argentina

Investigational Site Number : 0320005

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Rosario, Santa Fe, Argentina

Investigational Site Number : 0320003

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Buenos Aires, Argentina

Investigational Site Number : 0320002

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Buenos Aires, Argentina

Investigational Site Number : 0320001

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Buenos Aires, Argentina

Investigational Site Number : 0320006

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Córdoba, Argentina

Investigational Site Number : 0320004

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Mendoza, Argentina

Inst de Medicina Integral Professor Fernando Figueira- Site Number : 0760006

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Recife, Pernambuco, Brazil

Hospital Ernesto Dornelles Site Number : 0760004

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Porto Alegre, Rio Grande Do Sul, Brazil

Hospital Ernesto Dornelles- Site Number : 0760004

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Porto Alegre, Rio Grande Do Sul, Brazil

Clinica de Alergia Martti Antila- Site Number : 0760002

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Sorocaba, São Paulo, Brazil

Hospital das Clinicas FMUSP- Site Number : 0760001

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São Paulo, Brazil

Investigational Site Number : 1240001

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Vancouver, British Columbia, Canada

Investigational Site Number : 1240007

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Hamilton, Ontario, Canada

Investigational Site Number : 1240006

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Montreal, Quebec, Canada

Investigational Site Number : 2500001

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Lille, France

Investigational Site Number : 2500004

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Nice, France

Investigational Site Number : 2500002

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Paris, France

Ospedale Pediatrico Bambino Gesù - Investigational Site Number : 3800002

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Rome, Roma, Italy

AOU Luigi Vanvitelli - Investigational Site Number : 3800004

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Naples, Italy

Fondazione IRCCS Policlinico San Matteo - Investigational Site Number : 3800001

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Pavia, Italy

Investigational Site Number : 4840001

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San Juan Del Río, Querétaro, Mexico

Investigational Site Number : 4840004

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Durango, Mexico

Investigational Site Number : 4840002

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Veracruz, Mexico

Investigational Site Number : 5280001

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Rotterdam, Netherlands

Investigational Site Number : 6160001

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Lodz, Lódzkie, Poland

Investigational Site Number : 6160004

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Tarnow, Poland

Investigational Site Number : 7240001

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Barcelona, Barcelona [Barcelona], Spain

Investigational Site Number : 7240002

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Sabadell, Castilla Y León, Spain

Investigational Site Number : 7240006

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Jerez de La Frontera, Cádiz, Spain

Investigational Site Number : 7240003

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Santiago de Compostela, Galicia [Galicia], Spain

Investigational Site Number : 7240005

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Madrid, Madrid, Comunidad De, Spain

Investigational Site Number : 8260002

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Leicester, Leicestershire, United Kingdom

Investigational Site Number : 8260003

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Birmingham, United Kingdom

Investigational Site Number : 8260004

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Bradford, United Kingdom

Investigational Site Number : 8260001

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London, United Kingdom

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